The Food and Drug Administration (FDA) has granted Orphan Drug designation to alvelestat (MPH-966) for the treatment of alpha-1 antitrypsin deficiency (AATD).

AATD is a rare, genetic disease caused by mutations in the SERPINA1 gene. The genetic changes result in a deficiency of alpha-1 antitrypsin, a protein produced in the liver that helps protect the lungs from damaging enzymes, specifically neutrophil elastase. The disease begins in early adult life and can lead to pulmonary emphysema.

By inhibiting neutrophil elastase, alvelestat is expected to protect AATD patients from further lung damage. The Company is currently evaluating alvelestat for the treatment of severe AATD in the proof-of-concept phase 2 ASTRAEUS trial ( Identifier: NCT03636347).

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“We believe that the Orphan Drug designation represents an important regulatory milestone, recognizing the significant and urgent unmet need for new therapies to address AATD and specifically AATD-LD,” said Dr Denise Scots-Knight, CEO of Mereo. “We look forward to providing an update on our program before the end of the year.”

Alvelestat is also being evaluated in phase 1b/2 studies for COVID-19 and bronchiolitis obliterans syndrome following allogeneic hematopoietic stem cell transplant.


Mereo BioPharma receives US Orphan Drug designation for alvelestat in the treatment of alpha-1 antitrypsin deficiency. News release. Mereo BioPharma Group plc. October 26, 2021.

This article originally appeared on MPR